Background and objectives: Cerebral palsy is the most common cause of dyskinesia in children and is not curable by generalized rehabilitation, pharmacotherapy, Chinese medicine, exercise therapy, or surgery. To date, several case reports have demonstrated that umbilical cord mesenchymal stem cells (UCMSCs)/neural stem cells (NSCs) have a therapeutic role in children with cerebral palsy; however, there has been no large-sample clinical trial to verify this. Therefore, there is a need to evaluate the safety and effectiveness of UCMSCs/NSCs for the treatment of cerebral palsy in children.
Design: A prospective randomized parallel-controlled trial.
Methods: One hundred and ten children with cerebral palsy who will receive treatment in the First Affiliated Hospital of Dalian Medical University, China, will be randomly divided into five groups (n = 22 per group): control, nasal transplantation of UCMSCs, lumbar puncture transplantation of UCMSCs, nasal transplantation of NSCs, and lumbar puncture transplantation of NSCs. Cell transplantation will be correspondingly conducted in the latter four groups, with at least 1 × 107 cells per session, for two sessions within 4 weeks as one course, for a total of two courses.
Outcome measures: Evaluations will be performed before cell treatment and at 1, 3, 6, 9, and 12 months after the completion of two treatment courses, including Gross Motor Function Measure (GMFM)-88, GMFM-66, Fine Motor Function Measure (FMFM), Modified Ashworth Scale, the Gesell Developmental Schedules, electroencephalogram examination and brain imaging examination. The primary outcome of this study is the overall objective response rate calculated on the basis of the changes in GMFM-88 total score and GMFM-66 reference percentile. The secondary outcomes of this study include the duration of response and progression-free survival based on the GMFM-88 total score change and GMFM-66 percentile change as well as overall survival, FMFM score, Modified Ashworth Scale score, the Gesell Developmental Schedules score, electroencephalogram examination and brain imaging examination.
Discussion: This study aims to verify the efficacy and safety of UCMSCs/NSCs transplantation for the treatment of cerebral palsy in children, providing experimental data to support UCMSCs/NSCs therapy for cerebral palsy in clinical practice.
Ethics and dissemination: Written informed consent will be given by legal guardians or authorized surrogates of children with cerebral palsy as well as donors or their legal guardians. Design of the trial was completed in November 2016, and registered with ClinicalTrials.gov in December 2016. Participant recruitment was initialized in March 2017, and is expected to last 2 years. Data collection and follow-up visit will end in June 2020, and data analysis will be completed in December 2020. The results of this study will be disseminated by publications in peer-reviewed journals.
Trial registration: This trial was registered in the ClinicalTrials.gov with registration No. NCT03005249 on December 26, 2016.